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Clinical Trial
. 2009 Sep 17;114(12):2393-400.
doi: 10.1182/blood-2009-03-211797. Epub 2009 Jun 29.

Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996)

Peter L Greenberg  1 , Zhuoxin Sun, Kenneth B Miller, John M Bennett, Martin S Tallman, Gordon Dewald, Elisabeth Paietta, Richard van der Jagt, Jessie Houston, Mary L Thomas, David Cella, Jacob M Rowe
Affiliations
Clinical Trial

Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996)

Peter L Greenberg et al. Blood. .

Abstract

This phase 3 prospective randomized trial evaluated the efficacy and long-term safety of erythropoietin (EPO) with or without granulocyte colony-stimulating factor plus supportive care (SC; n = 53) versus SC alone (n = 57) for the treatment of anemic patients with lower-risk myelodysplastic syndromes. The response rates in the EPO versus SC alone arms were 36% versus 9.6%, respectively, at the initial treatment step, 47% in the EPO arm, including subsequent steps. Responding patients had significantly lower serum EPO levels (45% vs 5% responses for levels < 200 mU/mL vs > or = 200 mU/mL) and improvement in multiple quality-of-life domains. With prolonged follow-up (median, 5.8 years), no differences were found in overall survival of patients in the EPO versus SC arms (median, 3.1 vs 2.6 years) or in the incidence of transformation to acute myeloid leukemia (7.5% and 10.5% patients, respectively). Increased survival was demonstrated for erythroid responders versus nonresponders (median, 5.5 vs 2.3 years). Flow cytometric analysis showed that the percentage of P-glycoprotein(+) CD34(+) marrow blasts was positively correlated with longer overall survival. In comparison with SC alone, patients receiving EPO with or without granulocyte colony-stimulating factor plus SC had improved erythroid responses, similar survival, and incidence of acute myeloid leukemia transformation.

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Figures

Figure 1
Figure 1
Overall survival of patients randomized to the supportive care alone (arm A, n = 57) and EPO/G-CSF (arm B, n = 53) arms. No statistically significant difference in overall survival between these 2 groups of patients was demonstrated (P = .28).
Figure 2
Figure 2
Forest plot of hazard ratios (EPO/SC) for overall survival related to treatment for clinical subgroups. A univariate Cox proportional hazard model, stratified on randomization features, estimated hazard ratios, and significance for overall survival. The horizontal lines provide the 95% confidence interval for the ratios. The dotted vertical line represents the overall hazard ratio (0.77).
Figure 3
Figure 3
Time to leukemic transformation of patients randomized to the SC alone (arm A, n = 57) and EPO/G-CSF (arm B, n = 53) arms. No statistically significant difference in time to transformation between these 2 groups of patients was demonstrated (P = .83).
Figure 4
Figure 4
Cumulative incidence of leukemic transformation, with death as a competing risk event. No statistically significant difference in time to transformation between these 2 groups of patients was demonstrated when the competing risk of death was included (P = .34, cumulative incidence analysis).
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Comment in

References

    1. Greenberg PL. The myelodysplastic syndromes. In: Hoffman R, Benz E, Shattil S, editors. Hematology: Basic Principles and Practice. 3rd Ed. New York, NY: Churchill Livingstone; 1999. pp. 1106–1129.
    1. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079–2088. - PubMed
    1. Greenberg PL, Attar E, Battiwalla M, et al. NCCN Practice Guidelines for Myelodysplastic Syndromes, Version 1. 2009. J Nat Comp Canc Network. 2008;6(1):902–925.
    1. Stone RM, Bernstein SH, Demetri G, et al. Treatment with recombinant human erythropoietin in patients with myelodysplastic syndromes. Leuk Res. 1994;18(10):769–776. - PubMed
    1. Rose EH, Abels RI, Nelson RA, et al. The use of r-HuEPO in the treatment of anaemia related to myelodysplasia (MDS). Br J Haematol. 1995;89(4):831–837. - PubMed

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